Achondroplasia clinical trials at UCSF
7 in progress, 1 open to eligible people
VIrtual STudy in Achondroplasia for the US (VISTA)
open to eligible people ages 0 months to 18 years
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected…
Oakland, California and other locations
BMN 111 in Children With Achondroplasia (ACH)
Sorry, in progress, not accepting new patients
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections …
Oakland, California and other locations
Infigratinib in Children and Adolescents With Achondroplasia
Sorry, in progress, not accepting new patients
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the …
San Francisco, California and other locations
BMN 111 in Children With Achondroplasia
Sorry, in progress, not accepting new patients
This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will…
Oakland, California and other locations
BMN 111 in Children With Achondroplasia
Sorry, in progress, not accepting new patients
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
Oakland, California and other locations
Infigratinib in Children With Achondroplasia (ACH)
Sorry, accepting new patients by invitation only
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a…
Oakland, California and other locations
Clinical Assessment Study in Children With Achondroplasia (ACH)
Sorry, in progress, not accepting new patients
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain,…
Oakland, California and other locations
Our lead scientists for Achondroplasia research studies include Hind Al Saif Paul Harmatz.
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